A local hospital is adding gene editing technology to its list of treatments.
Children’s Health in Dallas is among a handful of hospitals in the nation that will soon begin treating sickle cell disease with gene therapy.
The FDA announced last month that it had approved two gene therapies, Casgevy and Lyfgenia, both of which are based on CRISPR technology, for use in patients 12 years old and older. These therapies are the first approved gene therapy treatments for the disease as previously reported by The Dallas Express.
Children’s Health already features a robust “Pediatric Sickle Cell Disease Program” which uses blood transfusions, bone marrow transplants, neurological care, and more.
“From the beginning, our pediatric sickle cell program pursued the development of innovative treatment approaches. For example, our hematologists spearheaded the use of treatments that are regularly applied today, resulting in the creation of a comprehensive set of diagnostic and management guidelines for children with sickle cell disease,” reads the hospital’s website.
The hospital will now be adding one of these new gene therapies to its list of treatments for the disease in children, reported Fox 4 KDFW.
Dr. Andrew Koh a pediatric hematologist and the director of the cellular and immunotherapeutics program at Children’s Health, is excited about the new gene therapies, explaining that the disease has a “major impact” on the nation.
“Essentially, you can take bone marrow-derived, or blood-derived stem cells, from the patient itself, genetically modify them to correct the sickle cell defect, and then re-infuse them back into the patient,” said Koh told Fox 4.
Koh said patients who take the treatment will know within a few months if it is working.
Crystal Dawn, whose three sons aged 10, 12, and 15 each suffer from the disease, said that she is excited about the prospect of the new therapy and wants to see if her children qualify for the treatment.
“Every day is a struggle. Every day is a battle,” said Dawn, per Fox 4.
Koh urges other patients to see if they qualify for the treatment.
